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Fear and pride as my son starts up with high school band
When I think about my son Charlie, I always remember my pregnancy with him. I’d […]
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US leadership emphasizes support for rare disease drug development
CureDuchenne is pleased that one of the main themes at the recent US FDA Cell […]
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Quest Podcast: Invisible People: Making the Rare Seen
In this Quest Podcast episode, we chat with a former pharmacist turned singer/songwriter who lives […]
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REGENXBIO Shares Positive Functional Data from Phase I/II AFFINITY DUCHENNE Trial of RGX-202
Today, REGENXBIO announced new data from its ongoing Phase I/II AFFINITY DUCHENNE® trial of RGX-202, […]
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PPMD’s Certified Care Center Program
PPMD continues to advocate to ensure families navigating a diagnosis of Duchenne and Becker muscular […]
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FDA grants orphan drug status to ISX9-CPC for heart issues in DMD
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to ISX9-CPC, IPS […]
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Regenxbio reports positive interim data from Phase 1/2 microdystrophin gene therapy trial
Regenxbio shared positive data from the AFFINITY DUCHENNE trial of RGX-202. In addition to robust […]
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Building empathy for Duchenne experiences through theater
Last Thursday, I participated in a workshop that used experiential theater to examine healthcare conversations. […]
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Pepgen pulls plug on exon-skipping therapy for Duchenne MD
Following lackluster data from a Phase 2 clinical trial, Pepgen will discontinue developing PGN-ED051, its experimental […]
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Steroids and Stress Dosing: Understanding the Updated PJ Nicholoff Steroid Protocol
Last week, PPMD brought together leading experts to address steroids, stress dosing and the updated […]
